First name
Ethan
Last name
Basch

Title

Benefits of Digital Symptom Monitoring With Patient-Reported Outcomes During Adjuvant Cancer Treatment.

Year of Publication

2021

Number of Pages

JCO2003375

Date Published

2021 Jan 28

ISSN Number

1527-7755

DOI

10.1200/JCO.20.03375

Alternate Title

J Clin Oncol

PMID

33507823

Title

Use of Patient-Reported Outcomes to Understand & Measure the Patient Experience of Novel Cell and Gene Therapies.

Year of Publication

2020

Date Published

2020 Jun 22

ISSN Number

2168-4804

Abstract

<p>Patient reported outcomes (PROs) are the gold standard for assessing patients' experience of treatment in oncology, defined in the 21st Century Cures Act as information about patients' experiences with a disease or condition, including the impact of a disease or condition, or a related therapy or clinical investigation on patients' lives; and patient preferences with respect to treatment of their disease or condition [1]. PROs provide a comprehensive assessment of the benefits and risks of new medical products, as well as essential data to inform real-world use. Although RCTs are the ultimate source for information for evaluating products in development, they are not always feasible for rare diseases with few or no effective treatment options available. Thus, it is important to consider other measures that can help to improve the strength of evidence for cell and gene therapies targeting rare indications. While collection of PROs and other patient experience endpoints does not resolve the difficulty of conducting trials in small populations, doing so contributes empirical evidence that informs both product development and patient access. Additionally, including routine collection of PROs in registries may provide supplemental data to further characterize the benefit:risk profile of cell and gene therapies at follow-up times that would be infeasible to operationalize in a clinical trial setting.</p>

DOI

10.1007/s43441-020-00184-6

Alternate Title

Ther Innov Regul Sci

PMID

32572771

Title

Reporting standards for patient-reported outcomes in clinical trial protocols and publications.

Year of Publication

2019

Date Published

2019 Apr 11

ISSN Number

1460-2105

DOI

10.1093/jnci/djz047

Alternate Title

J. Natl. Cancer Inst.

PMID

30959517

Title

Symptom Monitoring in Pediatric Oncology Using Patient-Reported Outcomes: Why, How, and Where Next.

Year of Publication

2017

Number of Pages

147-53

Date Published

2018 Apr

ISSN Number

1178-1661

Abstract

<p>Symptom monitoring using patient-reported outcomes (PROs) is not common in pediatric oncology, despite interest from stakeholders-including patients, families, clinicians, and regulatory organizations-and proven clinical benefit in adult oncology. This article examines the foundational data for patient-reported symptom reporting in this population and posits the next investigative steps toward the implementation of patient-reported symptom monitoring in the care and research of pediatric oncology patients. The reasoning behind, and feasibility of, monitoring symptoms in pediatric oncology patients using PRO measures are discussed, as well as specific tools that have been developed to track symptoms in this population, including innovative electronic self-reporting platforms built to engage children in the symptom reporting process. Aspects of engaging both patients and clinicians in the symptom self-report process are reviewed, as are the experiences of "early adopters" of this process in pediatric oncology and across pediatrics. It is clear that there are key issues that remain regarding the use of PROs for symptom monitoring, including selection of specific outcomes to monitor, how to resolve discrepant reports, and determination of benefit. The next steps for investigation of these issues are discussed. Unanswered questions notwithstanding, work should continue to make patient-reported symptom monitoring an established, evidence-based part of routine and research practice in pediatric oncology.</p>

DOI

10.1007/s40271-017-0279-z

Alternate Title

Patient

PMID

29071524

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